Fact Sheet: Breakthrough Therapies

On July 9, 2012 the Food and Drug Administration Safety and Innovation Act (FDASIA) was signed. FDASIA Section 902 provides for a new designation - Breakthrough Therapy Designation. A breakthrough therapy is a drug:

intended alone or in combination with one or more other drugs to treat a serious or life threatening disease or condition and

preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.

If a drug is designated as breakthrough therapy, FDA will expedite the development and review of such drug. All requests for breakthrough therapy designation will be reviewed within 60 days of receipt, and FDA will either grant or deny the request.

Please refer to section 902 of FDASIA for more specific information about this provision. Additionally, a Guidance for Industry: Expedited Programs for Serious Conditions––Drugs and Biologics was published on May 30, 2014, providing a single resource for information on FDA’s policies and procedures as well as threshold criteria generally applicable to conclude that a drug is a candidate for one or more of the expedited development and review programs: fast track designation, breakthrough therapy designation, accelerated approval, and priority review designation. .

Contact Information:

Center for Drug Evaluation and Research (CDER)

If your application is reviewed in CDER the CDER Breakthrough Therapy Program Manager is: